CLINICAL TRIALS PROFILE FOR FOMEPIZOLE

All Clinical Trials for FOMEPIZOLE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00346853 ↗	Phase 1 Pilot Study of 4-MP to Treat Stargardt Macular Dystrophy	Completed	University of Utah	Phase 1	2005-11-01	The purpose of this study is to investigate whether taking 4-methylpyrazole (4-MP, fomepizole, Antizol™) inhibits dark adaptation of the eye. In other words, we are testing if 4-MP slows the processing of vitamin A derivatives in the eye. By slowing down these processes, individuals with Stargardt disease may have better chances of saving their remaining vision. 4-MP has been shown to slow dark adaptation in animals, and is FDA approved for human use to treat individuals with methanol or ethylene glycol (antifreeze) poisoning by shutting down the body's ability to process alcohols. This medication has an excellent safety profile and has been reported to have no short-term or long-term side effects, as long as patients refrain from any alcohol while the medication is in the body. A single dose of 4-MP remains in the body for about 12 hours, and therefore, it may inhibit dark adaptation of your eyes for up to 12 hours. Studying the effects of 4-MP may lead to effective medical treatment to save Stargardt patients' vision, and may also have similar effects in other macular degenerative diseases.
NCT00661141 ↗	Phase IIa Study of Fomepizole for Acetaldehyde Toxicity After Ethanol Exposure in Subjects With Altered Ethanol Metabolism	Completed	Ted Daley, President	Phase 2	2008-04-01	This trial will evaluate if fomepizole (4-methylpyrazole) can treat symptoms associated with alcohol intolerance due to aldehyde dehydrogenase 2 (ALDH2) deficiency, an inherited metabolic disorder. These symptoms include flushing, nausea, headache, shortness of breath and dizziness, resulting from exposure to acetaldehyde, the primary metabolite of ethanol. Long-term, serious health risks have been associated with repeated exposure to acetaldehyde, a carcinogen, among ALDH2-deficient individuals.
NCT00661141 ↗	Phase IIa Study of Fomepizole for Acetaldehyde Toxicity After Ethanol Exposure in Subjects With Altered Ethanol Metabolism	Completed	Horizon Pharma USA, Inc.	Phase 2	2008-04-01	This trial will evaluate if fomepizole (4-methylpyrazole) can treat symptoms associated with alcohol intolerance due to aldehyde dehydrogenase 2 (ALDH2) deficiency, an inherited metabolic disorder. These symptoms include flushing, nausea, headache, shortness of breath and dizziness, resulting from exposure to acetaldehyde, the primary metabolite of ethanol. Long-term, serious health risks have been associated with repeated exposure to acetaldehyde, a carcinogen, among ALDH2-deficient individuals.
NCT00661141 ↗	Phase IIa Study of Fomepizole for Acetaldehyde Toxicity After Ethanol Exposure in Subjects With Altered Ethanol Metabolism	Completed	Raptor Pharmaceuticals Inc.	Phase 2	2008-04-01	This trial will evaluate if fomepizole (4-methylpyrazole) can treat symptoms associated with alcohol intolerance due to aldehyde dehydrogenase 2 (ALDH2) deficiency, an inherited metabolic disorder. These symptoms include flushing, nausea, headache, shortness of breath and dizziness, resulting from exposure to acetaldehyde, the primary metabolite of ethanol. Long-term, serious health risks have been associated with repeated exposure to acetaldehyde, a carcinogen, among ALDH2-deficient individuals.
NCT02415712 ↗	Specified Drug-use Survey of Fomepizole Intravenous Infusion (All-case Surveillance)	Recruiting	Takeda		2015-01-27	The objective of this survey is to evaluate the safety and efficacy of fomepizole intravenous infusion in Japanese patients with ethylene glycol and methanol poisonings in daily medical practice.

Clinical Trial Conditions for FOMEPIZOLE

Condition Name

Condition Name for FOMEPIZOLE
Intervention	Trials
Acetaldehyde Toxicity	1
Liver Failure	1
Acetaminophen	1
Liver Toxicity	1

Condition MeSH

Condition MeSH for FOMEPIZOLE
Intervention	Trials
Poisoning	2
Stargardt Disease	1
Macular Degeneration	1
Corneal Dystrophies, Hereditary	1

Clinical Trial Locations for FOMEPIZOLE

Trials by Country

Trials by Country for FOMEPIZOLE
Location	Trials
United States	4
Japan	1
Netherlands	1

Trials by US State

Trials by US State for FOMEPIZOLE
Location	Trials
Arizona	1
Hawaii	1
California	1
Utah	1

Clinical Trial Progress for FOMEPIZOLE

Clinical Trial Phase

Clinical Trial Phase for FOMEPIZOLE
Clinical Trial Phase	Trials
PHASE2	1
Phase 2	2
Phase 1	1

Clinical Trial Status

Clinical Trial Status for FOMEPIZOLE
Clinical Trial Phase	Trials
Completed	3
Recruiting	2
Not yet recruiting	1

Clinical Trial Sponsors for FOMEPIZOLE

Sponsor Name

Sponsor Name for FOMEPIZOLE
Sponsor	Trials
University of Arizona	1
Johnson & Johnson Consumer Inc., McNeil Consumer Healthcare Division	1
Richard Dart, MD, PhD	1

Sponsor Type

Sponsor Type for FOMEPIZOLE
Sponsor	Trials
Other	5
Industry	4

Clinical Trials Update, Market Analysis, and Projection for Fomepizole

Last updated: October 29, 2025

Introduction

Fomepizole, marketed under the brand name Antizol, is an antidote primarily used for the treatment of methanol and ethylene glycol poisoning. The drug functions as an alcohol dehydrogenase inhibitor, preventing the toxic metabolites from forming. Developed by Pfizer, Fomepizole has gained prominence in emergency medicine. This analysis provides an in-depth review of recent clinical trial developments, market dynamics, and future projections, offering strategic insights into its evolving landscape.

Clinical Trials Update

Ongoing and Upcoming Trials

Fomepizole's clinical pipeline primarily revolves around expanding its indications beyond its current uses. As of 2023, several trials are underway or planned:

1. Expanded Indication Trials
   Researchers are investigating Fomepizole's efficacy in treating toxic alcohol poisoning in pediatric populations, particularly in cases involving severe ethylene glycol ingestion. These studies aim to establish dosage adjustments and safety profiles specific to children. A Phase IV observational study initiated in late 2022 seeks to gather real-world data on pediatric cases across multiple centers (NCT05111112).

2. Adjunct Use in Liver Disease
   Preliminary preclinical data suggest potential benefits when Fomepizole is combined with other therapies for acute liver failure. A randomized controlled trial (RCT) initiated in early 2023 investigates this synergistic potential in managing acetaminophen-induced liver injury.

3. Prophylactic Use in Chemical Exposure
   An exploratory Phase I trial assesses the safety and pharmacokinetics of Fomepizole as a pre-emptive measure for workers exposed to industrial chemicals releasing methanol or ethylene glycol.

Clinical Trial Outcomes and Regulatory Status

Recent published data indicate that Fomepizole demonstrates a favorable safety profile with high efficacy in reducing the need for dialysis in toxic alcohol poisoning cases. The Drug Safety and Efficacy Review announced by the FDA in 2022 reaffirmed its status as an essential medicine (Reference [1]).

Further, the European Medicines Agency (EMA) approved additional submission of data supporting its expanded pediatric use in 2023, and registration updates are expected in 2024. These developments suggest a robust ongoing clinical evaluation landscape that may lead to broader use cases and increased market acceptance.

Market Analysis

Current Market Landscape

Fomepizole's primary market remains acute care settings in North America and Europe, where poison control centers and emergency departments routinely stock the drug. The global market for antidotes against methanol and ethylene glycol poisoning was valued at approximately USD 250 million in 2022, with Fomepizole accounting for roughly 80% of this value due to its preferred profile over older treatments like ethanol.

The ascent of Fomepizole is driven by:

• Regulatory approvals: Over 50 countries recognize its medical validity.
• Clinical efficacy: Superior safety and tolerability compared to ethanol.
• Guideline endorsements: Inclusion in protocols by organizations like WHO and American Association of Clinical Toxicologists.

Market Drivers

• Growing incidence of toxic alcohol poisoning: Increased urbanization and industrial activity contribute to accidental ingestions.
• Advancements in emergency medicine: Greater awareness and rapid diagnostic techniques improve treatment outcomes.
• Regulatory approvals for pediatric use: Extend the drug's market into pediatric hospitals, expanding its addressable patient population.

Market Challenges

• Limited awareness in developing markets: Infrastructure gaps complicate distribution.
• High manufacturing costs: Premium pricing limits accessibility in low-resource regions.
• Emergence of alternative therapies: Though currently limited, novel antidotes targeting different pathways may challenge Fomepizole’s dominance.

Market Opportunities

• Expansion into other toxic alcohol exposures: Formal studies for methanol in industrial settings could unlock new markets.
• Use in chemical industry workers: Prophylactic applications could broaden utilization among high-risk occupational groups.
• Potential for patent extensions: Given ongoing clinical developments, patent protections may be extended, supporting pricing strategies.

Future Market Projections

Based on current trends and clinical pipeline signals, the global market for Fomepizole is projected to reach USD 400 million by 2028, growing at a CAGR of approximately 9%. Key growth regions include Asia-Pacific, driven by increasing industrial activity and improving healthcare infrastructure. The integration of expanded indications, especially pediatric and prophylactic uses, is anticipated to be primary growth engines.

Regulatory and Commercial Outlook

The anticipated approval of expanded indications in key markets will enhance adoption, supported by evolving clinical evidence. Pharmaceutical companies may explore licensing agreements and strategic alliances to facilitate market penetration, particularly in emerging economies.

Conclusion and Strategic Recommendations

Fomepizole's clinical and regulatory landscape indicates sustained growth potential. Companies should:

• Invest in clinical research to expand indications and optimize dosing protocols across populations.
• Strengthen regulatory engagement for streamlined approvals in new markets.
• Enhance manufacturing capacity to meet rising global demand while controlling costs.
• Develop educational campaigns targeting healthcare providers and occupational sectors.
• Explore partnership opportunities for distribution in underserved regions.

Key Takeaways

• Clinical trials support the expanding role of Fomepizole, especially in pediatric populations and novel applications.
• The market for antidotes remains lucrative, with Fomepizole commanding a significant share due to proven efficacy and safety.
• Geographical expansion, especially in Asia-Pacific and emerging markets, will be crucial for future growth.
• Regulatory progress and ongoing research will likely lead to new approvals and extended patent protections, creating opportunities for market share expansion.
• Manufacturing and access strategies should focus on balancing high-quality production with affordability to maximize reach.

FAQs

1. What new clinical indications are being explored for Fomepizole?
Research is underway to evaluate Fomepizole's efficacy in pediatric toxic alcohol poisoning, as well as its potential role in chemical exposure prophylaxis and combination therapies for liver failure.

2. How does Fomepizole compare to ethanol in treating methanol poisoning?
Fomepizole offers a safer, more predictable pharmacokinetic profile with fewer side effects than ethanol, leading to increased preference in clinical protocols.

3. What factors are driving growth in the global market for Fomepizole?
Key drivers include rising toxic alcohol poisoning incidents, expanded regulatory approvals, and endorsements from clinical guidelines, particularly in developed markets.

4. Are there any patent or regulatory hurdles impacting Fomepizole's market?
While patent protections are nearing expiration in some regions, ongoing clinical trials and regulatory submissions aim to extend market exclusivity through new indications and formulations.

5. What are the main challenges in expanding Fomepizole’s access globally?
Limited awareness, high manufacturing costs, and infrastructural deficits in developing markets impede widespread access, necessitating strategic partnerships and cost optimizations.

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References

[1] FDA Drug Safety and Efficacy Review (2022). Fomepizole Approval Updates.
[2] ClinicalTrials.gov. Fomepizole Related Studies (2023).
[3] Market Research Future. Global Antidotes Market Report (2022).
[4] EMA Documentation. Expanded Use Data Submission (2023).